FDA Lifts Clinical Hold on Neurizon's ALS Drug, Paving Way for Human Trials

The U.S. Food and Drug Administration (FDA) has lifted a clinical hold on Neurizon's investigational drug for amyotrophic lateral sclerosis (ALS), allowing the Melbourne-based biotech company to proceed with human trials. The decision comes after months of delays and additional data submissions, highlighting both the challenges in ALS drug development and the current strains on regulatory processes.

Clinical Hold Lifted After Extended Review

Neurizon announced on Monday that the FDA has removed the clinical hold placed on the investigational new drug (IND) application for NUZ-001, the company's candidate for ALS and other neurodegenerative diseases. The hold, initially imposed in February, required additional preclinical animal exposure studies to be conducted.

Despite submitting a complete response letter with new preclinical data from rat and dog studies in July, Neurizon faced unexpected delays in the FDA's review process. The company attributed these delays to broader issues within the agency, citing "agency-wide restructuring and staffing reductions under recent administrative reforms" as factors impacting the FDA's ability to maintain timely review cycles.

Path Forward for NUZ-001

With the clinical hold now lifted, Neurizon is set to advance NUZ-001 into human trials. The drug will be studied as part of the HEALEY ALS Platform Trial, a privately funded adaptive trial hosted by Massachusetts General Hospital. Neurizon expects the hospital to file a protocol amendment to its IND, with patient enrollment targeted to begin in the fourth quarter of 2025.

Michael Thurn, CEO of Neurizon, expressed gratitude to key opinion leaders and patient advocacy groups, stating, "Their efforts in raising awareness and lobbying for the urgent needs of the ALS community have been invaluable in helping us reach this point."

Challenges in ALS Drug Development

The progress of NUZ-001 comes against a backdrop of significant challenges in ALS drug development. The HEALEY ALS Platform Trial, established in 2020, has seen numerous setbacks, with investigational drugs from companies such as Denali, AbbVie, and Calico failing to show efficacy. To date, only Prilenia Therapeutics and Clene have managed to produce enough positive biomarker data through the HEALEY trial to advance their molecules.

These difficulties underscore the complexities involved in developing effective treatments for ALS, a devastating neurodegenerative disease that continues to pose significant challenges to researchers and pharmaceutical companies alike.